A Phase 2 interventional study of Pomalidomide Oral Product and Placebo oral capsule in Telangiectasia, Hereditary Hemorrhagic, sponsored by The Cleveland Clinic. Completed at 14 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-23.
Sponsored by The Cleveland Clinic · Phase 2, Interventional, and Treatment
This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.
HHT is associated with substantial morbidity, leading to a reduced quality of life, decreased rate of employment and a high incidence of depression. There currently exists no medical therapy recognized as consistently efficacious in HHT. Reports of the efficacy of thalidomide in HHT, as well as interim results of a pilot trial of pomalidomide in HHT provide evidence of efficacy with minimal toxicity. The favorable efficacy:toxicity ratio of pomalidomide suggest that it may benefit patients with HHT.
This study is designed as a Phase II placebo-controlled double-blind study of pomalidomide in HHT patients with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks.
Primary Objective: To determine efficacy of pomalidomide compared to placebo for the reduction in severity of epistaxis after 24 weeks of treatment.
Secondary Objectives: To determine the safety and tolerability of pomalidomide for the treatment of HHT; to determine if pomalidomide treatment improves quality of life in HHT; to determine whether a continued response to pomalidomide is evident 4 weeks after treatment discontinuation; to develop a biorepository for future studies to define biomarkers predictive of pomalidomide response and allow investigations into the biology of HHT and mechanisms of pomalidomide.
106 studies on the registry are indexed under Telangiectasia, Hereditary Hemorrhagic; 24 are open to participants now.
This study's enrollment of 145 is above the median of 33 across 61 interventional studies indexed under Telangiectasia, Hereditary Hemorrhagic.
Browse Telangiectasia, Hereditary Hemorrhagic studies →The Cleveland Clinic is the lead sponsor of 831 studies on the registry; 119 are open to participants now.
Of its 90 completed or terminated interventional studies of FDA-regulated products, 73 (81%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Anything that in the investigator's opinion is likely to interfere with completion of the study
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Oral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.
Drug: Pomalidomide Oral Product
A placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks.
Drug: Placebo oral capsule
Pomalidomide, a third generation derivative of thalidomide, given orally at a starting dose of 4 mg/day for days 1-28 of six 28-day cycles. The dose may be reduced to 3 or 2 mg/day based on specific adverse event (AE) criteria.
Also known as: POMALYST
Matching placebo will be given.
Change From Baseline Epistaxis Severity Score
The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
Time frame: 4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment
Average Total Daily Duration of Nosebleeds - Change From Baseline
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Weighted Average Total Daily Duration of Nosebleeds - Change From Baseline
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary, weighted by the intensity, with 90%% winsorization. The outcome is the change between the baseline diary on the specified timepoint
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no infusions have a value of zero.
Time frame: Baseline through 24 Weeks
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Time frame: Baseline through 12 Weeks
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Time frame: 12 through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 24 Weeks
Patients with any packed red blood cells transfusion through the 24-week treatment period
Time frame: Baseline through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 12 Weeks
Patients with any packed red blood cells transfusion through the first 12 weeks of the treatment period
Time frame: Baseline through 12 Weeks
Patients With Any Packed Red Blood Cells Transfusion 12-24 Weeks
Patients with any packed red blood cells transfusion through the second 12 weeks of the treatment period
Time frame: 12 through 24 Weeks
Neuro-QoL - Satisfaction With Social Roles and Activities - T Score
The outcome measure is the Neuro-QoL Satisfaction with Social Roles and Activities T-Score to compare the outcomes of Pomalidomide versus Placebo. The Neuro-QoL Satisfaction with Social Roles and Activities Short Form (V1.1) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more satisfaction. The minimal detectable change is 3.7 T score points
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Patient Reported Outcomes Measurement Information System (PROMIS) - Emotional Distress - Depression - T Score
The outcome measure is the PROMIS Emotional Distress - Depression T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS Emotional Distress-Depression Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more depression
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
PROMIS - Fatigue - T Score
The outcome measure is the PROMIS Fatigue T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS® Fatigue Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more fatigue
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
HHT-Specific QOL Questionnaire - Score
The outcome measure is the HHT-Specific QOL Questionnaire - Score to compare the outcomes of Pomalidomide versus Placebo. The HHT-specific QOL score ranges from 0 to 16 with higher scores indicating more limitations due to HHT in the prior 4 weeks
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Average Total Daily Duration of Low Intensity Nosebleeds - Change From Baseline
The daily of low intensity epistaxis duration is calculated as the total duration of "spotting" or "dripping" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Average Total Daily Duration of Medium Intensity Nosebleeds - Change From Baseline
The daily of medium intensity epistaxis duration is calculated as the total duration of "dripping quickly" or "steady stream" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Average Total Daily Duration of High Intensity Nosebleeds - Change From Baseline
The daily of high intensity epistaxis duration is calculated as the total duration of "gushing" or "pouring" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Total Blood Transfused
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no transfusions have a value of zero.
Time frame: Baseline through 24 Weeks
Total Blood Transfused
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no transfusions have a value of zero.
Time frame: Baseline through 12 Weeks
Total Blood Transfused
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no transfusions have a value of zero.
Time frame: 12 through 24 Weeks
Transferrin Saturation
Change from baseline transferrin saturation collected from blood iron studies
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Ferritin
Change from baseline ferritin level collected from blood iron studies
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Hemoglobin
Change from baseline Hemoglobin level taken from complete blood counts
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Hematocrit
Change from baseline Hematocrit level taken from complete blood counts
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Mean Corpuscular Volume (MCV)
Change from baseline MCV level taken from complete blood counts
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Mean Corpuscular Hemoglobin Concentration (MCHC)
Change from baseline MCHC level taken from complete blood counts
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
Platelets
Change from baseline platelets taken from complete blood counts
Time frame: After 4, 8, 12, 16, 20 and 24 weeks of treatment, and 4 weeks post-treatment
| Milestone | Pomalidomide | Placebo |
|---|---|---|
| Started | 95 | 49 |
| Completed | 63 | 41 |
| Not completed | 32 | 8 |
| Withdrew: Adverse event | 14 | 0 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 2 |
| Withdrew: Joint withdrawal decision | 1 | 0 |
| Withdrew: Study terminated by sponsor | 7 | 4 |
| Withdrew: Physician decision | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
| Withdrew: Withdrawal by subject | 7 | 2 |
The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -1 (-1.40 to -0.60) | -1.1 (-1.50 to -0.70) |
| 8 Weeks | -1.3 (-1.80 to -0.70) | -1.2 (-1.60 to -0.80) |
| 12 Weeks | -1.3 (-1.90 to -0.80) | -1.6 (-2.00 to -1.20) |
| 16 Weeks | -1.2 (-1.90 to -0.60) | -2 (-2.40 to -1.60) |
| 20 Weeks | -1.3 (-1.90 to -0.60) | -2 (-2.40 to -1.50) |
| 24 Weeks | -1.2 (-1.90 to -0.50) | -2.1 (-2.60 to -1.50) |
| 4 Weeks Post-treatmenr | -0.7 (-1.30 to 0.00) | -1.6 (-2.10 to -1.20) |
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
| minutes | Placebo | Pomalidomide |
|---|---|---|
| 12 Weeks | -4 (-8.40 to 0.50) | -6.7 (-10.40 to -3.00) |
| 24 Weeks | -4.4 (-10.50 to 1.70) | -5.3 (-11.00 to 0.50) |
| 4 Weeks Post-treatment | -1.7 (-7.50 to 4.20) | -4.6 (-7.90 to -1.30) |
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary, weighted by the intensity, with 90%% winsorization. The outcome is the change between the baseline diary on the specified timepoint
| minutes | Placebo | Pomalidomide |
|---|---|---|
| 12 Weeks | -4.3 (-10.40 to 1.70) | -12.1 (-16.60 to -7.60) |
| 24 Weeks | -4 (-13.50 to 5.60) | -11.5 (-16.60 to -6.30) |
| 4 Weeks Post-treatment | -2.6 (-10.60 to 5.40) | -9.3 (-14.10 to -4.60) |
The daily of low intensity epistaxis duration is calculated as the total duration of "spotting" or "dripping" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
| minutes | Placebo | Pomalidomide |
|---|---|---|
| 12 Weeks | -2 (-5.20 to 1.30) | -1.6 (-2.90 to -0.20) |
| 24 Weeks | -2.8 (-6.10 to 0.50) | -1.6 (-2.70 to -0.40) |
| 4 Weeks Post-treatment | -1.8 (-5.00 to 1.30) | -0.6 (-2.00 to 0.80) |
The daily of medium intensity epistaxis duration is calculated as the total duration of "dripping quickly" or "steady stream" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
| minutes | Placebo | Pomalidomide |
|---|---|---|
| 12 Weeks | -1.9 (-4.10 to 0.40) | -3.6 (-6.00 to -1.30) |
| 24 Weeks | -3.6 (-8.70 to 1.60) | -2.2 (-7.00 to 2.70) |
| 4 Weeks Post-treatment | -2 (-6.30 to 2.40) | -2.5 (-4.90 to 0.00) |
The daily of high intensity epistaxis duration is calculated as the total duration of "gushing" or "pouring" nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
| minutes | Placebo | Pomalidomide |
|---|---|---|
| 12 Weeks | -0.1 (-1.50 to 1.20) | -1.4 (-2.60 to -0.20) |
| 24 Weeks | 1.9 (-2.70 to 6.50) | -1.5 (-2.80 to -0.10) |
| 4 Weeks Post-treatment | 2 (-3.90 to 7.90) | -1.6 (-3.30 to 0.10) |
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no infusions have a value of zero.
| mg/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Iron Infused | 204 (33 to 341) | 170 (0 to 408) |
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no infusions have a value of zero.
| mg/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Iron Infused | 170 (0 to 340) | 225 (0 to 510) |
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no infusions have a value of zero.
| mg/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Iron Infused | 333.3 (0 to 500) | 0 (0 to 340) |
Patients with any packed red blood cells transfusion through the 24-week treatment period
| Participants | Placebo | Pomalidomide |
|---|---|---|
| No transfusion | 38 | 77 |
| Yes transfusion | 11 | 15 |
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no transfusions have a value of zero.
| units of blood/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Blood Transfused | 0 (0.00 to 0.00) | 0 (0.00 to 0.00) |
Patients with any packed red blood cells transfusion through the first 12 weeks of the treatment period
| Participants | Placebo | Pomalidomide |
|---|---|---|
| No transfusion | 43 | 79 |
| Yes transfusion | 6 | 13 |
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no transfusions have a value of zero.
| units of blood/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Blood Transfused | 0 (0.00 to 0.00) | 0 (0.00 to 0.00) |
Patients with any packed red blood cells transfusion through the second 12 weeks of the treatment period
| Participants | Placebo | Pomalidomide |
|---|---|---|
| No transfusion | 37 | 67 |
| Yes transfusion | 8 | 7 |
Total blood transfused (units of blood) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no transfusions have a value of zero.
| units of blood/4 weeks | Placebo | Pomalidomide |
|---|---|---|
| Total Blood Transfused | 0 (0.00 to 0.00) | 0 (0.00 to 0.00) |
Change from baseline transferrin saturation collected from blood iron studies
| Percent | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | 8.2 (-7.20 to 23.60) | -3.7 (-8.70 to 1.20) |
| 8 Weeks | 4.4 (-4.10 to 12.80) | -1.1 (-6.60 to 4.40) |
| 12 Weeks | -2.3 (-7.90 to 3.40) | 9 (-2.50 to 20.60) |
| 16 Weeks | 3.8 (-4.70 to 12.30) | 3.3 (-2.60 to 9.20) |
| 20 Weeks | 6.2 (-5.30 to 17.80) | 1.5 (-3.80 to 6.80) |
| 24 Weeks | 10.1 (-0.30 to 20.40) | 22.7 (-17.50 to 62.90) |
| 4 Weeks Post-treatment | 12.8 (-9.40 to 35.10) | 1.2 (-4.20 to 6.50) |
Change from baseline ferritin level collected from blood iron studies
| ng/mL | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -25.5 (-48.50 to -2.60) | -6.5 (-42.30 to 29.40) |
| 8 Weeks | 11.5 (-28.40 to 51.30) | 2.6 (-45.40 to 50.60) |
| 12 Weeks | -12.4 (-48.10 to 23.30) | 2.5 (-32.20 to 37.10) |
| 16 Weeks | 13.1 (-35.40 to 61.50) | 8.2 (-32.10 to 48.40) |
| 20 Weeks | 31.6 (-24.70 to 87.90) | -7.4 (-46.30 to 31.50) |
| 24 Weeks | 47.4 (-12.30 to 107.10) | -5.4 (-41.00 to 30.20) |
| 4 Weeks Post-treatment | -10.9 (-49.30 to 27.40) | -18.5 (-59.80 to 22.90) |
Change from baseline Hemoglobin level taken from complete blood counts
| g/dL | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -0.2 (-0.50 to 0.20) | -0.4 (-0.70 to -0.10) |
| 8 Weeks | -0.3 (-0.70 to 0.10) | -0.3 (-0.60 to 0.10) |
| 12 Weeks | 0 (-0.60 to 0.60) | 0 (-0.40 to 0.40) |
| 16 Weeks | -0.2 (-0.90 to 0.40) | 0.4 (-0.10 to 0.80) |
| 20 Weeks | 0 (-0.60 to 0.70) | 0.4 (-0.20 to 0.90) |
| 24 Weeks | -0.1 (-0.60 to 0.50) | 0.3 (-0.20 to 0.90) |
| 4 Weeks Post-treatment | -0.5 (-1.20 to 0.20) | 0.7 (0.10 to 1.20) |
Change from baseline Hematocrit level taken from complete blood counts
| Percent | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -0.3 (-1.40 to 0.80) | -1.4 (-2.30 to -0.40) |
| 8 Weeks | -0.7 (-1.90 to 0.50) | -0.9 (-2.10 to 0.30) |
| 12 Weeks | 0.1 (-1.40 to 1.60) | -0.2 (-1.50 to 1.10) |
| 16 Weeks | -0.6 (-2.40 to 1.20) | 0.4 (-1.00 to 1.70) |
| 20 Weeks | -0.3 (-2.00 to 1.40) | 0.2 (-1.30 to 1.70) |
| 24 Weeks | -0.7 (-2.30 to 0.90) | 0.3 (-1.30 to 1.90) |
| 4 Weeks Post-treatment | -1.6 (-3.50 to 0.30) | 1 (-0.40 to 2.40) |
Change from baseline MCV level taken from complete blood counts
| fL | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -0.1 (-1.10 to 0.80) | -0.4 (-1.30 to 0.40) |
| 8 Weeks | 0 (-1.10 to 1.10) | -1.2 (-2.30 to -0.20) |
| 12 Weeks | 0.1 (-1.30 to 1.40) | 0.1 (-1.00 to 1.20) |
| 16 Weeks | -0.2 (-2.00 to 1.60) | 0.9 (-0.40 to 2.10) |
| 20 Weeks | 1.3 (-0.60 to 3.20) | 2.2 (0.70 to 3.60) |
| 24 Weeks | 1.6 (-0.50 to 3.70) | 3.2 (1.60 to 4.80) |
| 4 Weeks Post-treatment | 0.2 (-1.80 to 2.30) | 3.2 (1.60 to 4.70) |
Change from baseline MCHC level taken from complete blood counts
| g/dL | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | -0.2 (-0.50 to 0.10) | 0 (-0.30 to 0.30) |
| 8 Weeks | -0.2 (-0.50 to 0.10) | 0.1 (-0.20 to 0.40) |
| 12 Weeks | 0 (-0.50 to 0.40) | 0.1 (-0.20 to 0.40) |
| 16 Weeks | -0.2 (-0.70 to 0.30) | 0.6 (0.30 to 1.00) |
| 20 Weeks | 0.3 (-0.30 to 0.90) | 0.7 (0.30 to 1.20) |
| 24 Weeks | 0 (-0.40 to 0.40) | 0.6 (0.20 to 1.10) |
| 4 Weeks Post-treatment | -0.2 (-0.70 to 0.30) | 0.9 (0.30 to 1.40) |
Change from baseline platelets taken from complete blood counts
| 10^3/uL platelets | Placebo | Pomalidomide |
|---|---|---|
| 4 Weeks | 7.8 (-5.70 to 21.30) | 13.4 (-8.00 to 34.90) |
| 8 Weeks | -2.3 (-17.40 to 12.70) | 19.3 (-0.60 to 39.20) |
| 12 Weeks | 4.9 (-12.90 to 22.70) | 0.9 (-18.20 to 20.10) |
| 16 Weeks | 10.3 (-15.90 to 36.50) | -19.7 (-36.90 to -2.50) |
| 20 Weeks | -2.1 (-18.10 to 13.90) | -24.1 (-46.10 to -2.10) |
| 24 Weeks | 4 (-11.50 to 19.50) | -27.3 (-51.50 to -3.00) |
| 4 Weeks Post-treatment | 5.7 (-11.60 to 22.90) | -13 (-33.00 to 7.00) |
The outcome measure is the Neuro-QoL Satisfaction with Social Roles and Activities T-Score to compare the outcomes of Pomalidomide versus Placebo. The Neuro-QoL Satisfaction with Social Roles and Activities Short Form (V1.1) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more satisfaction. The minimal detectable change is 3.7 T score points
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Baseline | 44.7 (42.9 to 46.4) | 45.5 (44.1 to 46.8) |
| 12 Weeks | 46.5 (44.6 to 48.4) | 47.5 (46.1 to 49.0) |
| 24 Weeks | 46 (43.7 to 48.2) | 47.6 (46.0 to 49.3) |
| 4 Weeks Post-treatment | 45.6 (43.0 to 48.3) | 48.8 (47.2 to 50.4) |
The outcome measure is the PROMIS Emotional Distress - Depression T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS Emotional Distress-Depression Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more depression
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Baseline | 0.2 (0.00 to 0.50) | -0.1 (-0.30 to 0.10) |
| 12 Weeks | 0 (-0.30 to 0.30) | -0.1 (-0.30 to 0.10) |
| 24 Weeks | -0.1 (-0.40 to 0.20) | -0.2 (-0.40 to 0.10) |
| 4 Weeks Post-treatment | 0.1 (-0.30 to 0.50) | -0.3 (-0.50 to -0.10) |
The outcome measure is the PROMIS Fatigue T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS® Fatigue Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more fatigue
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Baseline | 57.7 (54.6 to 60.8) | 55.7 (53.6 to 57.8) |
| 12 Weeks | 55.8 (53.2 to 58.3) | 54.7 (52.5 to 56.9) |
| 24 Weeks | 56.2 (52.9 to 59.6) | 54.2 (51.6 to 56.9) |
| 4 Weeks Post-treatment | 59.5 (56.0 to 63.1) | 52.3 (50.1 to 54.5) |
The outcome measure is the HHT-Specific QOL Questionnaire - Score to compare the outcomes of Pomalidomide versus Placebo. The HHT-specific QOL score ranges from 0 to 16 with higher scores indicating more limitations due to HHT in the prior 4 weeks
| units on a scale | Placebo | Pomalidomide |
|---|---|---|
| Baseline | 7.1 (6.10 to 8.10) | 5.8 (5.20 to 6.40) |
| 12 Weeks | 5 (3.90 to 6.10) | 3.5 (2.70 to 4.30) |
| 24 Weeks | 5.6 (4.40 to 6.80) | 3.6 (2.80 to 4.30) |
| 4 Weeks Post-treatment | 5.9 (4.50 to 7.40) | 3.7 (2.90 to 4.40) |
Collected over 6 Months. Non-serious events are listed at a 0.00% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 0/49 (0%) | 8/49 (16.3%) | 43/49 (87.8%) |
| Pomalidomide | 1/95 (1.1%) | 22/95 (23.2%) | 90/95 (94.7%) |
| Event | Placebo | Pomalidomide |
|---|---|---|
| Gastrointestinal haemorrhageGastrointestinal disorders | 2/49 | 1/95 |
| PneumoniaInfections and infestations | 2/49 | 2/95 |
| AnaemiaBlood and lymphatic system disorders | 0/49 | 2/95 |
| Iron deficiency anaemiaBlood and lymphatic system disorders | 0/49 | 2/95 |
| Cardiac failure congestiveCardiac disorders | 0/49 | 2/95 |
| SyncopeNervous system disorders | 0/49 | 2/95 |
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 0/49 | 2/95 |
| Haemorrhagic anaemiaBlood and lymphatic system disorders | 1/49 | 1/95 |
| Diverticulum intestinal haemorrhagicGastrointestinal disorders | 1/49 | 0/95 |
| Portal vein thrombosisHepatobiliary disorders | 1/49 | 0/95 |
| Event | Placebo | Pomalidomide |
|---|---|---|
| ConstipationGastrointestinal disorders | 9/49 | 45/95 |
| FatigueGeneral disorders | 17/49 | 42/95 |
| NeutropeniaBlood and lymphatic system disorders | 5/49 | 41/95 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 5/49 | 22/95 |
| RashSkin and subcutaneous tissue disorders | 2/49 | 20/95 |
| Corona virus infectionInfections and infestations | 9/49 | 9/95 |
| DizzinessNervous system disorders | 7/49 | 16/95 |
| NauseaGastrointestinal disorders | 8/49 | 15/95 |
| Oedema peripheralGeneral disorders | 1/49 | 14/95 |
| DiarrhoeaGastrointestinal disorders | 7/49 | 7/95 |
| Age, Continuous(years) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Mean | 58.7 ± 11 | 58.8 ± 13 | 58.8 ± 12 |
| Sex/Gender, Customized(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Female | 24 | 45 | 69 |
| Male | 25 | 50 | 75 |
| Race/Ethnicity, Customized(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Asian | 1 | 0 | 1 |
| Black/African American | 5 | 6 | 11 |
| Other | 0 | 3 | 3 |
| Unknown | 2 | 1 | 3 |
| White | 41 | 85 | 126 |
| Race/Ethnicity, Customized(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Hispanic/Latino | 3 | 2 | 5 |
| Not Hispanic/Latino | 44 | 90 | 134 |
| Unknown/Not Reported | 2 | 3 | 5 |
| Marital status(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Divorced | 3 | 10 | 13 |
| Domestic Partner | 0 | 2 | 2 |
| Married | 33 | 64 | 97 |
| Never Been Married | 9 | 15 | 24 |
| Separated | 2 | 1 | 3 |
| Unknown | 0 | 1 | 1 |
| Widowed | 2 | 2 | 4 |
| Pre-menopausal(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| No | 17 | 33 | 50 |
| Not Applicable | 25 | 50 | 75 |
| Yes | 7 | 12 | 19 |
| Education(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Associate degree | 5 | 8 | 13 |
| Bachelor�s degree | 12 | 15 | 27 |
| Doctoral degree | 1 | 5 | 6 |
| Don�t Know | 18 | 36 | 54 |
| High School Grad | 4 | 9 | 13 |
| Master�s degree | 4 | 9 | 13 |
| Refused | 1 | 3 | 4 |
| Years of college no degree (specify) | 4 | 9 | 13 |
| grade (specify) | 0 | 1 | 1 |
| Employment status(Participants) | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Disabled permanently or temporary | 3 | 2 | 5 |
| Looking for work unemployed | 0 | 1 | 1 |
| Other (specify) | 9 | 16 | 25 |
| Retired | 12 | 28 | 40 |
| Working Now | 25 | 48 | 73 |
12 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — The Data Coordinating Center (DCC) will prepare de-identified data files for sharing. A final data sharing plan will be developed with NHLBI and the study Steering Committee (SC). The data can be provided in Statistical Analysis System (SAS) data sets and export files and documentation will be in portable document format (PDF). The first level of data sharing will be with trial investigators. In addition, the DCC will be responsible for sharing study data with outside researchers, if approved by NHLBI and the SC.
Supporting information: Study protocol, Statistical analysis plan
Time frame: Data will be made available to investigators 6 months after closed data sets for the trial have been prepared for final analysis or 1 month after results of the protocol are published in a peer-reviewed journal.
Access criteria: A data request form will be developed to collect information deemed necessary by the SC and NIH, including applicant name, organization, and purpose of research. Investigators will submit the request form, Data Distribution Agreement, and Institutional Review Board (IRB) approval to designated DCC staff. The SC or its designated Committee will review each application, and if the requestor meets established criteria for access to the data and provides the required documents, the requested data sets and associated documentation will be disseminated by a mode agreed upon by the SC in accordance with NHLBI policy.
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Studies of the drug tested here: Pomalidomide
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